Abstract
Checkpoint inhibitors have transformed the treatment of cancer in adults. This class of drugs has demonstrated encouraging results in various malignancies such as metastatic melanoma, bladder cancer, renal cancer, and non-small cell lung carcinoma. However, researchers have only begun investigating the effectiveness and tolerability of checkpoint inhibitors in pediatric patients. We conducted a review of PubMed indexed literature and clinicaltrials.gov using combinations of the keywords checkpoint, inhibitor, pediatric, CTLA-4 (cytotoxic T lymphocyte antigen-4), PD-1 (programmed cell death-1), and PD-L1 (programmed cell death receptor-1 ligand) to find every recently completed and ongoing trial evaluating checkpoint inhibitors in patients younger than 21 years old. Pertinent articles and clinical trials discussing the role of immune checkpoint inhibitors in the pediatric population were selected for final analysis and manuscript citation. This review presents an overview of the cellular mechanisms involved in checkpoint inhibition and of studies evaluating checkpoint inhibitors in humans. The review also details results and side effects from studies conducted with pediatric patients, current pediatric clinical trials, and future implications. Immune checkpoint inhibitors have the potential to further therapeutic advances in pediatric oncology; however, we need more clinical trials and combination drug strategies targeted toward pediatric cancers.
Highlights
Checkpoint inhibitors are among the most researched anticancer agents of the 21st century
We conducted a review of PubMed indexed literature and clinicaltrials.gov using combinations of the keywords checkpoint, inhibitor, pediatric, cytotoxic T lymphocyte antigen-4 (CTLA-4), PD-1, and programmed cell death receptor-1 ligand (PD-L1) to find every recently completed and ongoing trial evaluating checkpoint inhibitors in patients younger than 21 years old
Given that most clinical trials to date have evaluated checkpoint inhibitors in adult patients, this review presents a brief summary of studies in humans of all age groups and focuses on results and side effects in pediatric patients
Summary
Checkpoint inhibitors have transformed the treatment of cancer in adults. This class of drugs has demonstrated encouraging results in various malignancies such as metastatic melanoma, bladder cancer, renal cancer, and non–small cell lung carcinoma. Researchers have only begun investigating the effectiveness and tolerability of checkpoint inhibitors in pediatric patients. Results: This review presents an overview of the cellular mechanisms involved in checkpoint inhibition and of studies evaluating checkpoint inhibitors in humans. The review details results and side effects from studies conducted with pediatric patients, current pediatric clinical trials, and future implications. Conclusion: Immune checkpoint inhibitors have the potential to further therapeutic advances in pediatric oncology; we need more clinical trials and combination drug strategies targeted toward pediatric cancers
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